Advanced Cell Technology (OTC BB: ACTC) EU’s 1st Human Embryonic Stem Cell Transplant in Patient with Stargardt’s Disease
ACTC.OB treated the first patient in its P1/2 clinical trial for Stargardt’s macular dystrophy (SMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs).
The P1/2 trial will involve 12 patients, with cohorts of 3 patients each in an ascending dosage format and is similar in design to the FDA-approved US trial that was initiated in 7/11.
The open-label study is designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation in patients with Stargardt’s macular dystrophy at 12 months, the study’s primary endpoint.
- The patient, who is severely sight-impaired, underwent transplantation of fully differentiated retinal pigment epithelial (RPE) cells derived from human embryonic stem cells. There were no complications, and the patient has tolerated the surgical procedure well;
- While this is primarily a safety trial, ACT will have the opportunity to image engraftment of RPE cells non-invasively and to assess any changes in sight;
- The surgery was performed on Friday, 1/20, at the Moorfields Eye Hospital in London. The patient successfully underwent the procedure without any complications;
- ACT and Moorfields had received clearance in September from theUK’s Medicines and Healthcare products Regulatory Agency (MHRA).
The Bottom Line: The treatment of the 1st patient in Europe is tangible evidence that stem cell research and development of cell therapies is making progress. It is also … milestone for scientists, stem cell advocates and patients hoping for cures. ACTC’s clinical trial program for hESC-derived RPE cells has taken another … critical step as they move forward with treating patients.